DADA2 diagnosed in adulthood versus childhood: A comparative study on 306 patients including a systematic literature review and 12 French cases

Semin Arthritis Rheum. 2021 Dec;51(6):1170-1179. doi: 10.1016/j.semarthrit.2021.09.001. Epub 2021 Sep 16.

Abstract

Background: Deficiency of adenosine deaminase 2 (DADA2) is a rare autoinflammatory disease usually presenting before the age of 10 years. Non-specific clinical features or late-onset presentation may delay its diagnosis until adulthood.

Objective: To determine whether DADA2 diagnosed in adulthood is associated with specific characteristics compared to DADA2 diagnosed in childhood.

Methods: We pooled a cohort of 12 adult DADA2 patients followed in France with cases identified through a systematic literature review. For each patient, we determined the type of clinical presentation and assessed six key organ involvements.

Results: A total of 306 cases were included. Among the 283 patients with available data regarding age at diagnosis, 140 were diagnosed during adulthood and 143 during childhood. The vascular presentation of DADA2 was more frequent in the adult diagnosis group (77.9% vs. 62.9%, p < 0.01), whereas the hematological presentation (bone marrow failure) prevailed in the pediatric diagnosis group (10.0% vs. 20.3% p = 0.02). In patients with vasculopathy, severe skin manifestations developed in 35% and 10% of the adult and pediatric diagnosis groups, respectively. Conversely, fewer strokes occurred in the adult group presenting with systemic vasculopathy (54% vs. 81%). Symptomatic humoral immune deficiency (HID) was rarely a clinical presentation in itself (5% and 2.8%) but accompanied other phenotypes of DADA2, especially the hematological phenotype in the adult group (33% vs. 4%).

Conclusion: DADA2 diagnosed in adulthood presents more often with a vascular phenotype and less often with bone marrow failure than DADA2 diagnosed in childhood. Adults diagnosed with DADA2 vasculopathy display more severe skin involvement but fewer strokes.

Keywords: Adenosine deaminase 2; Bone marrow failure disorders; DADA2; Polyarteritis nodosa; Pure red-cell aplasia; Vasculitis.

Publication types

  • Systematic Review

MeSH terms

  • Adenosine Deaminase* / genetics
  • Adult
  • Child
  • Humans
  • Immunologic Deficiency Syndromes*
  • Intercellular Signaling Peptides and Proteins / genetics
  • Mutation
  • Phenotype

Substances

  • Intercellular Signaling Peptides and Proteins
  • Adenosine Deaminase